ArticleEMBO reports2002
Functional human CFTR produced by a stable minichromosome.
Article in EMBO reports, 2002. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed.
- DNA Methylation Patterns Correlate with the Expression ofInternational journal of molecular sciences · 2021Article
- On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis.Frontiers in pharmacology · 2021Review
- Efficient size-independent chromosome delivery from yeast to cultured cell lines.Nucleic acids research · 2017Article
- Generation of a conditionally self-eliminating HAC gene delivery vector through incorporation of a tTAVP64 expression cassette.Nucleic acids research · 2015Article
- A pathway from chromosome transfer to engineering resulting in human and mouse artificial chromosomes for a variety of applications to bio-medical challenges.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2015Review
- De novo formed satellite DNA-based mammalian artificial chromosomes and their possible applications.Chromosome research : an international journal on the molecular, supramolecular and evolutionary aspects of chromosome biology · 2015Review
- Re-engineering an alphoid(tetO)-HAC-based vector to enable high-throughput analyses of gene function.Nucleic acids research · 2013Article
- A new generation of human artificial chromosomes for functional genomics and gene therapy.Cellular and molecular life sciences : CMLS · 2013Review
- Organization of synthetic alphoid DNA array in human artificial chromosome (HAC) with a conditional centromere.ACS synthetic biology · 2012Article
- Human artificial chromosomes for gene delivery and the development of animal models.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
- CFTR expression and activity from the human CFTR locus in BAC vectors, with regulatory regions, isolated by a single-step procedure.Gene therapy · 2010Article
- CFTR expression from a BAC carrying the complete human gene and associated regulatory elements.Journal of cellular and molecular medicine · 2009Article
- Genetically modified pigs produced with a nonviral episomal vector.Proceedings of the National Academy of Sciences of the United States of America · 2006Article
- Genetic modification of hematopoietic stem cells with nonviral systems: past progress and future prospects.Gene therapy · 2005Review
- Artificial and engineered chromosomes: developments and prospects for gene therapy.Chromosoma · 2005Review
- The manipulation of chromosomes by mankind: the uses of microcell-mediated chromosome transfer.Chromosoma · 2005Review
- Gene therapy progress and prospects: episomally maintained self-replicating systems.Gene therapy · 2004Review
- Reconstructing large regions of an ancestral mammalian genome in silico.Genome research · 2004Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
Abstract
Artificial chromosomes have been claimed to be the ideal vector for gene therapy, but their use has been hampered by an inability to produce stable and well designed molecules. We have used a structurally defined minichromosome to clone the human cystic fybrosis transmembrane conductance regulator (CFTR) locus. To guarantee the presence of the proper regulatory elements, we used the 320 kb yeast artificial chromosome (YAC) 37AB12 with the intact CFTR gene and upstream sequences. The resulting minichromosome was analyzed for the presence of the entire CFTR gene and for its functional activity by molecular and functional methods. We have identified clones showing the presence of both the transcript and the CFTR protein. Moreover, in the same clones, a chloride secretory response to cAMP was detected. Mitotic and molecular stability after prolonged growth without selection demonstrated that the constructs were stable. This is the first example of a structurally known minichromosome made to contain an active therapeutic gene.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.